Consensus guidelines for the optimal management of adverse events in newly diagnosed, transplant-ineligible patients receiving melphalan and prednisone in combination with thalidomide (MPT) for the treatment of multiple myeloma.

Thalidomide has received approval from the European Agency for the Evaluation of Medicinal Products for the treatment of newly diagnosed multiple myeloma (MM) patients older than 65 years or ineligible for transplant. The results of five phase III trials assessing thalidomide in combination with melphalan and prednisone (MPT) have demonstrated significantly improved response rates compared with melphalan and prednisone (MP) alone. Additionally, two of these studies showed that survival was extended by approximately 18 months in patients treated with MPT compared with MP alone. Thalidomide, in combination with MP, is associated with adverse events (AEs) including peripheral neuropathy and venous thromboembolism. In order to optimize the efficacy of MPT, a good awareness of these AEs is imperative. This manuscript outlines both evidence- and consensus-based recommendations discussed by a panel of experts, to provide a practical guide for physicians addressing the effective management of newly diagnosed, transplant-ineligible MM patients receiving thalidomide therapy.

Congenital amegakaryocytic thrombocytopenia--report of a new c-mpl gene missense mutation.

A 44-month old girl with congenital amegakaryocytic thrombocytopenia, already with pancytopenia, underwent an unrelated allogeneic cord blood transplantation with recovery of normal blood cell counts. The patient was a compound heterozygote for two c-mpl missense mutations inherited from both parents, one of them, a G578A exon 4 mutation leading to a cysteine to tyrosine replacement of codon 193, previously unreported.

Large-volume leukaphereses may be more efficient than standard-volume leukaphereses for collection of peripheral blood progenitor cells.

To overcome the need for multiple leukaphereses to collect enough PBPC for autologous transplantation, large-volume leukaphereses (LVL) are used to process multiple blood volumes per session. We compared the efficiency of CD34+ cell collection by LVL (n = 63; median blood volumes processed 11.1) with that of standard-volume leukaphereses (SVL) (n = 38; median blood volumes processed 1.9). To achieve this in patients with different peripheral blood concentrations of CD34+ cells, we analyzed the ratio of CD34+ cells collected per unit of blood volume processed, divided by the number of CD34+ cells in total blood volume at the beginning of apheresis. For LVL, 30% (9%-323%) of circulating CD34+ cells were collected per blood volume compared with 42% (7%-144%) for SVL (p = 0.02). However, in LVL patients, peripheral blood CD34+ cells/L decreased a median of 54% during LVL (similar data for SVL not available). The number of CD34+ cells collected per blood volume processed after 4 and 8 blood volumes and at the end of LVL were 0.32 (0.01-2.05), 0.24 (0.01-1.68), and 0.22 (0.01-2.40) x 10(6) CD34+ cells/kg, respectively (p = 0.0007), despite the 54% decrease in peripheral blood CD34+ cells/L throughout LVL. A median 66% decrease in the platelet count was also observed during LVL. Thus, LVL may be more efficient than SVL for PBPC collection, allowing, in most patients, the collection in one LVL of sufficient PBPC to support autologous transplantation.